Beacon Therapeutics has released interim nine-month results from its Phase 2/3 DAWN trial and 36-month follow-up data from its Phase 2 SKYLINE trial evaluating Laru-zova in patients with X-linked retinitis pigmentosa (XLRP).
The company said that 43 patients were enrolled in the DAWN trial, with 28 receiving Laru-zova. Interim results indicated improvements in retinal sensitivity, functional vision, and vision-related quality of life measures compared to baseline. According to the company, the treatment was well tolerated, with no dose-limiting toxicities reported.
In the SKYLINE study, which followed 16 patients over 36 months, the company claims that improvements in retinal sensitivity and visual function observed at 12 months were maintained through three years. No new safety concerns were reported during the extended follow-up.
Beacon Therapeutics said that these findings support the potential of Laru-zova as a one-time gene therapy for patients with XLRP, a rare inherited retinal disease that can cause progressive vision loss and blindness. The company added that it plans to continue advancing the program and engaging with regulatory authorities.
